The FDA's new pathway for rare disease drugs, based on causal biology, is scientifically promising. However, the name "plausible mechanism" is a critical flaw. The term sounds weak, creating doubt for patients and giving payers powerful leverage to deny coverage by implying a lower standard of evidence.
"China Speed," once synonymous with rapid antibody development, now extends to RNA silencing technologies. A surge in homegrown RNAi companies and programs, with dozens unpartnered, indicates China's biotech ecosystem is rapidly diversifying into new, complex therapeutic modalities beyond its established strengths.
An innovative strategy for solid tumors involves using bispecific T-cell engagers to target the tumor stroma—the protective fibrotic tissue surrounding the tumor. This novel approach aims to first eliminate this physical barrier, making the cancer cells themselves more vulnerable to subsequent immune attack.
Richard Pazdur's immediate goal as the new CDER director is to restore stability and integrity at the FDA. His initial focus will be on rebuilding the team by recruiting, retaining, and empowering staff—deferring major policy shifts like accelerated approval reform until the agency's morale and operational capacity are restored.
Initial panic over the MFN drug pricing scheme was based on pegging U.S. prices to the lowest in the industrialized world. The actual proposal is far less drastic, targeting the second-lowest price among a small cohort of high-income nations (G7 plus Denmark and Switzerland), a significantly less onerous benchmark.
To combat immunosuppressive "cold" tumors, new trispecific antibodies are emerging. Unlike standard T-cell engagers that only provide the primary CD3 activation signal, these drugs also deliver the crucial co-stimulatory signal (e.g., via CD28), ensuring full T-cell activation in microenvironments where this second signal is naturally absent.